Promise and Progress - Another Breakthrough Treatment for Leukemia

Sidney Kimmel Gives Hopkins Its Biggest Gift Ever

Another Breakthrough Treatment for Leukemia

Date: December 1, 2002

A new gene-targeted therapy promises to do for acute myeloid leukemia (AML) what Gleevec did for chronic myeloid leukemia (CML). Hopkins investigators hope it will transform AML, the most common form of adult leukemia, from one of the most lethal types of leukemia to one of the most treatable.

The new therapy focuses on a specific subtype of AML referred to as FLT3-positive. Right now, less than 10 percent of people with this form of AML are cured, but this new therapy could help most of them.

The discovery centers around a compound, technically known as a tyrosine kinase inhibitor, that targets FLT3 (pronounced: flit three) gene mutations, a biological mistake that occurs in more than 30 percent of AML cases, leading to a more aggressive, treatment-resistant form of the disease. Researchers are testing a drug in the laboratory that interferes with abnormal FLT3 function. “By blocking the action of the altered gene, we can render it powerless. It’s as if the mutation no longer exists,” says Donald Small, M.D., Ph.D., associate professor of oncology, the first to clone the FLT3 gene and director of this research. “This is the payoff for more than a decade of laboratory research to pinpoint the genetic alterations associated with this type of leukemia. Now, we have turned it around so that the very abnormalities that cause the disease to progress are now a part of the cure.”